MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.

What this Trial Is About

Researchers are evaluating a phase II trial of a reduced intensity conditioned allogeneic hematopoietic cell transplant (HCT) using post-transplant cyclophosphamide (PTCy) for patients with idiopathic severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH), acquired pure red cell aplasia (aPRCA), or acquired amegakaryocytic thrombocytopenia (aAT). The study uses population pharmacokinetic-guided dosing for conditioning and adjusts low dose total body irradiation based on age and presence of myelodysplasia or clonal hematopoiesis. Participants are divided into two groups: those 25 years or younger without clonal hematopoiesis, and those aged 25 to 75 years and/or with clonal hematopoiesis. Each group receives a conditioning regimen followed by stem cell infusion and graft-versus-host disease (GvHD) prevention with drugs including rituximab if needed, rabbit ATG, cyclophosphamide, fludarabine, total body irradiation, tacrolimus, and mycophenolate mofetil. The study treatment phase includes conditioning through day +180, followed by supportive care and follow-up lasting two years post-transplant. During the study, participants undergo regular assessments including monitoring for acute and chronic GvHD, blood cell recovery, and survival up to two years after transplant. Treatment adherence and safety are closely tracked, and various outcomes such as incidence of GvHD and failure-free survival are measured at one and two years post-HCT. This comprehensive follow-up helps evaluate the effects and safety of the transplant approach in these blood disorders.

MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.